In this project, we will be using our human induced pluripotent stem cell (hiPSC)-derived in vitro model system for MFS to develop new therapies aimed at ameliorating the disease phenotype. Our approaches include correcting the mutations in FBN1, the gene coding for fibrillin-1, identifying targets downstream of dysfunctional FBN1 implicated in MFS pathogenesis, and transitioning our novel therapies into more physiologically relevant in vitro culture and in vivo model systems.